Long-Term Outcomes of Hematopoietic Stem Cell Transplantation for Severe Treatment-Resistant Autoimmune Cytopenia in Children

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Quality of Survivorship in a Rare Disease: Clinicofunctional Outcome and Physical Activity in an Observational Cohort Study of 618 Long-Term Survivors of Ewing Sarcoma

PurposeSignificantly improved survival rates in patients with Ewing sarcoma have raised interest in accessing the quality of long-term survivorship. In this study, subjective and objective measurement tools, preclassified as physical or mental scores, were used to assess clinicofunctional outcome and physical activity after intensive bone tumor treatment.MethodsLong-term outcome of 618 survivors from consecutive Ewing sarcoma trials was assessed by the Toronto Extremity Salvage Score, Short-Form Health Survey (SF-36), Brief Symptom Inventory (BSI), and Rosenberg Self-Esteem Scale questionnaires and by the accelerometric StepWatch 3 Activity Monitor. Prospective measurements were correlated retrospectively with standardized primary trial data. Results were compared with 316 nonrandom healthy peers by using effect sizes (d). Median observation time was 12.9 years from primary diagnosis (range, 3.7 to 31.2 years).ResultsAbsolute subjective scores were moderate to good for survivors. Compared with control subjects, unfavorable outcome was shown on physical Toronto Extremity Salvage Score, SF-36 Physical Component Summary, and BSI-Somatization scales (|d| 0.50; P <.01), in contrast to SF-36 Mental Component Summary, BSI-Anxiety, BSI-Depression, and Rosenberg Self-Esteem Scale mental scales (|d| 0.31). Survivors were less active than control subjects, as demonstrated by a step count difference of 1,742 steps per day (d = -0.43; P <.01); however, on average, the recommended level for an active lifestyle was achieved ( 10,000 steps). Location of pelvic tumor was the major inferior disease-specific prognostic factor in physical scores (P <.01), whereas nondisease-specific inferior factors in questionnaires were older age and female sex (P <.01).ConclusionSurvivors of Ewing sarcoma apparently returned to a normal life with minor limitations. Observed reductions in physical scores should be a focus in future research to optimize treatment strategies to reduce a negative impact on the quality of survivorshi

Oryza australiensis leaf transcriptome

De novo transcriptome assembly of Oryza australiensis based on leaf RNAseq data (SRA SRR1178926). Trinity v2.4.0 was used for assembly using default parameters, with Trimmomatic for trimming of reads before assembly.<br

Long-term outcomes of hematopoietic stem cell transplantation for severe treatment-resistant autoimmune cytopenia in children

AbstractWe analyzed the long-term outcomes of pediatric patients registered in the European Group for Blood and Marrow Transplantation database who underwent hematopoietic stem cell transplantation (HSCT) for severe treatment refractory autoimmune cytopenia. With a median follow-up of 100 months, event-free survival was 54% overall, with no significant difference between allogeneic HSCT (n = 15) and autologous HSCT (n = 7) recipients (58% versus 42%; P = .50). Despite a trend toward failure of response or relapse after autologous HSCT compared with allogeneic HSCT, the difference was not significant (43% versus 13%; P = .30). Treatment-related mortality was high in both HSCT groups (29% and 16%; P = .09). Based on the limited numbers of subjects in this retrospective analysis, both allogeneic and autologous HSCT may induce complete and persistent responses in approximately one-half of pediatric patients with severe refractory autoimmune cytopenia, although treatment-related toxicity is high